Question:

If the results of initial testing on a drug are amazing – and could mean the difference between life and death – should pharmaceutical companies be required to go through the current long and costly process of drug testing before making it available to the public? 

If a drug is fine-tuned to treat a specific genetic profile, does it make sense to do randomized controlled trials, even if it means delaying the availability of the drug and possibly increasing the cost, thereby rendering it unattainable to certain patients?

The Renewed Debate over Drug Testing

Two drugs that were recently approved by the United States Food and Drug Administration (FDA) have helped renew this debate. In 2011, Zelboraf, a drug that targets specific mutations in cancer cells, was approved by the FDA after an early-stage trial showed eight out of ten patients experienced significant tumor shrinkage.  Earlier this year, another drug – Tafinlar, a drug targeting the same mutant genes as Zelboraf – was approved by the FDA based on a single clinical trial of just 250 patients.

Tafinlar is available at 30 percent less the cost of Zelboraf.

Are Randomized Controlled Trials Becoming Obsolete?

For many years, randomized controlled trials have been the most effective way of seeing if a drug works.  However, according to Richard Pazdur, M.D., the FDA expert who wanted to shorten the Zelboraf trial, “The types of drugs that we’re seeing now are different.  They are just simply better in terms of efficacy.”    Scientists have a better understanding of molecular changes that promote cancer growth.  For example, an estimated 50-60 percent of melanoma patients – the people Zelboraf and Tafinlar target – have a specific genetic mutation. For drugs that focus on patients with specific genetic mutations, randomized controlled trials may not make sense anymore.

Will Faster Drug Approval Impact Pricing?

Eleven of the twelve cancer drugs launched in the United States last year cost more than $100,000 a year per patient.  In April, more than 100 leukemia specialists from around the world spoke up in the American Society of Hematology’s journal Blood that cancer drug prices were “too high, unsustainable, may compromise access of needy patents to highly effective therapy, and are harmful to the sustainability of our national healthcare systems.”

Accounting for 36 percent of the total research expenditure in 2012, clinical trials are the biggest single cost in drug company R&D.  Many contend that if researchers were permitted to test certain treatments on just people with a specific targeted mutation, researchers could work more quickly, and with fewer patients, to determine if the treatment is effective – bringing these treatments to patients more quickly and, possibly, more cheaply.  Unfortunately, there has not been a significant decrease in medications with the most recent changes in testing procedures.  Paul Workman, head of drug discovery for Britain’s Institute of Cancer Research says, “The costs should be coming down tremendously.  What’s disappointing is that we haven’t seen it happen yet.”

European Drug Regulators Vs. FDA

Part of the issue is that European drug regulators and particularly government funding agencies, such as the National Institute for Health and Clincial Excellence (NICE), may not be willing to accept products based on the FDA’s more flexible clinical trial standards.  In Europe, even if a drug is approved by the European regulatory agencies, if the governments don’t approve funding for it, it will still not be available to many people.

It will be interesting to see what happens.  As Workman said, “We are in a fascinating but frustrating period of transition.”

This post is based on Insight: How new cancer drugs can skip randomized trials by Julie Steenhuysen and Ben Hirschler published in Reuters on September 26, 2013.